Scientists are preparing to monitor the increasing number of patients undergoing an unproven surgical treatment for the human form of BSE, but will not include them in official trials without more evidence of its potential and government approval.

The father of the first patient to receive pentosan polysulphate two years ago believes that it has slowed or even stopped the disease's progression, and it is understood similar treatment is now being given to two other variant CJD patients.

But pentosan has to be pumped round the brain and requires neurosurgery. There are uncertainties over dosage and how frequently it needs to be given, despite what its supporters see as promising results from animal trials.

The drug has blood-thinning properties, prompting concerns over brain haemorrhages, and two group of government advisers have so far refused to approve formal evaluation of the technique.

But scientists are anxious not to alienate families who have chosen the radical and controversial treatment, and are offering them the chance to speak to a neurologist who could collate information and systematically monitor the effects of the drug.

Don Simms, whose son Jonathan has vCJD, won a legal battle to get pentosan through the NHS. He said: "It is not a wonder drug. It is not a cure. I am convinced we have slowed the disease down or we have stopped it, one or the other."

Meanwhile nearly 30 patients have been enrolled on trials of a tablet-based drug called quinacrine, formerly used to combat malaria and sometimes still used against arthritis. These trials, overseen by the Medical Research Council, started in the summer nearly three years after the government asked the MRC to fasttrack research into what then appeared to be the frontrunner in vCJD treatments.

That request followed the experience of Rachel Forber, a British vCJD patient who went to the US to try quinacrine but subsequently died. The government has now asked scientists to monitor pentosan patients, but without sanctioning official trials.

John Collinge, head of the MRC's prion unit, said it was up to individual families if they wanted to take up the offer: "I am very sympathetic to families. I am trying to help them." But the trial had been set up specifically for quinacrine, which had needed ethical approval, peer review and monitoring by a committee with independent members.

Not all scientists support the quinacrine trial but Professor Collinge said it was far too early to say the drug would not work, despite the difficulties in tracking progress across a range of diseases linked to the abnormal form of the prion protein. "No treatment like this is going to reverse damage," Prof Collinge said. "The most we can expect of these first-generation drugs is they slow down or at best stop the damage to the brain."

The quinacrine trial was expected to last three years and several dozen patients were still needed to garner statistically reliable results from what was thankfully a relatively rare group of diseases. "You cannot just treat four patients and say it works or it doesn't work."

http://www.guardian.co.uk/uk_news/story/0,3604,1373806,00.html

> Meanwhile nearly 30 patients have been enrolled on trials of a > tablet-based drug called quinacrine, formerly used to combat malaria > and sometimes still used against arthritis. These trials, overseen by > the Medical Research Council, started in the summer nearly three years > after the government asked the MRC to fasttrack research into what > then appeared to be the frontrunner in vCJD treatments.>

Neurology -- Future Table of Contents Alert

A new future TOC for Neurology has been made available for the issue:28 December 2004; Vol. 63, No. 12

URL: http://www.neurology.org/future/63.12.shtml

snip...

Compassionate use of quinacrine in Creutzfeldt-Jakob disease fails to show significant effects